Challenges in the use of highly effective modulator treatment for cystic fibrosis.
Challenges in the use of highly effective modulator treatment for cystic fibrosis.
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DOI：
10.1016/j.jcf.2021.01.007
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发表时间：
2021-05
期刊：
影响因子：
--
通讯作者：
Taylor-Cousar JL
中科院分区：
文献类型：
--
作者：
Ramos KJ;Pilewski JM;Taylor-Cousar JL
在过去的十年里，口服小分子疗法的发展，解决了基本的囊性纤维化跨膜传导调节因子（CFTR）蛋白缺陷。高效调节剂治疗（HEMT）对绝大多数囊性纤维化（CF）患者有效，有望改变这种慢性限制生命的疾病的现状。一些CF患者有CFTR基因型，使他们有资格进行HEMT，但也有合共病，使他们被排除在导致美国食品和药物管理局批准的最初的III期临床试验之外。本综述的目的是探讨HEMT在具有挑战性的情况下的应用，包括晚期CF肺病患者、实体器官移植后、妊娠期间和CF相关疾病患者的应用。
The last decade has seen development of oral, small molecule therapies that address the basic cystic fibrosis transmembrane conductance regulator (CFTR) protein defect. Highly effective modulator treatment (HEMT) that is efficacious for a large majority of people living with cystic fibrosis (CF) promises to change the landscape of this chronic life-limiting disease. Some people living with CF have a CFTR genotype that renders them eligible for HEMT, but also have comorbidities that excluded them from the original Phase III clinical trials that led to US Food and Drug Administration approval. The purpose of this review is to address the use of HEMT in challenging situations, including initiation for those with advanced CF lung disease, and use after solid organ transplant, during pregnancy, and for individuals with CFTR-related disorders without a definitive diagnosis of CF.
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影响因子：
24.3
作者：
De Muynck, Benedicte;Van Herck, Anke;Vos, Robin
通讯作者：
Vos, Robin
影响因子：
168.9
作者：
Heijerman, Harry G. M.;McKone, Edward F.;Mccoy, Karen S.
通讯作者：
Mccoy, Karen S.
影响因子：
8.9
作者：
Chambers, Daniel C.;Cherikh, Wida S.;Stehlik, Josef
通讯作者：
Stehlik, Josef
DOI：
10.1016/j.jcf.2016.07.011
发表时间：
2017-01
期刊：
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
影响因子：
--
作者：
Fink AK;Yanik EL;Marshall BC;Wilschanski M;Lynch CF;Austin AA;Copeland G;Safaeian M;Engels EA
通讯作者：
Engels EA
影响因子：
2.1
作者：
Berastegui, Cristina;Gomez-Olles, Susana;Roman, Antonio
通讯作者：
Roman, Antonio